This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle…
This research study is being done to compare the combination of lenalidomide and dexamethasone followed by autologous peripheral blood stemcell transplant (PBSCT) and lenalidomide and dexamethasone without PBSCT in subjects with untreated multiple myeloma. This comparison will include how many…
This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stemcells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent…
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem…
The purpose of this study is to see if Isatuximab can alter the immune system favorably in patients with multiple myeloma or lymphoma upon recovery from the autologous stemcell transplantation. This study will have two arms. On one arm (control arm), Participants…
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stemcell transplant from a family member or unrelated adult donor for a non-malignant condition.
The purpose of this study is to find out whether treatment with daratumumab and lenalidomide will make your Multiple Myeloma better. There are 2 treatment groups in this study. You will either receive lenalidomide alone or both lenalidomide and daratumumab along with medications to take before and after the daratumumab injection.
The purpose of this study is to determine if the investigational therapy called bb2121 is effective in treating multiple myeloma. bb2121 is a type of therapy known as chimeric antigen receptor (CAR) T-cell therapy. T cells are a kind of white blood cell which form…
This is a research study for subjects that have been previously treated for systemic light chain (AL)amyloidosis (a disease in which abnormal protein deposits can damage organs and tissues in your body) and nowrequire further treatment. The main purpose of this study is to evaluate the safety and determine the maximum…
The purpose of this research study is to explore the drug combination, lenalidomide, bortezomib, and dexamethasone alone or when combined with autologous stemcell transplantation to see what side effects it may have and how well it works for treatment of newly diagnosed multiple myeloma.
This is a Phase 2 study for patients with myelofibrosis previously treated with one or more Janus Kinase(JAK) Inhibitor. A JAK inhibitor targets a certain pathway that may be causing cancer to grow, bystopping or inhibiting the cycle of development of the pathway. There is one JAK inhibitor that has beenapproved for the treatment of patients with…